During the last three years the development of gene therapy has been rapid. The number of gene therapies on the market has more than doubled and within certain disease areas, such as hemophilia, a large proportion of patients will likely be successfully treated. The implementation will be demanding for the Swedish health care system, owing to the very high cost of these drugs, but total costs may be reduced, especially if a single treatment is sufficient. New disruptive technologies such as gene editing are being applied in clinical trials in disease areas such as hemoglobinopathies.
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